Clinical trials: Genetic Diseases
Metachromatic Leukodystrophy (MLD)
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).
- Trial name
- IDEAMLD (NCT01510028)
- Testing
- Recombinant human arylsulfatase A
- Where
- 5 sites in Australia, Denmark and 3 more
- Volunteers
- 24 people
- Sponsor
- Shire
Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with…
- Trial name
- NCT04307381
- Testing
- Donidalorsen
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Geographic Atrophy, Stargardt Disease
Phase 1 Study of C.001 in Retinal Degeneration
This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.
- Conditions
- Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
- Trial name
- NCT07594236
- Testing
- Injection
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cellio Therapeutics Inc
Atopic Dermatitis
A Study to Evaluate the Effectiveness and Safety of Topical OPA-15406 Ointment to Treat Participants With Atopic Dermatitis
The purpose of this study is to investigate the effectiveness and safety of 2 concentrations of OPA-15406 compared to vehicle in participants with atopic dermatitis (AD).
- Trial name
- NCT02068352
- Testing
- OPA-15406, Vehicle ointment
- Where
- 30 sites in United States, Australia and 1 more
- Volunteers
- 121 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Primary Immunodeficiency Diseases (PID)
Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN)
This study will provide insights on the infusion parameters, dosing, and experience of participants transitioning to CUVITRU in a real-world setting.
- Trial name
- NCT03716700
- Testing
- CUVITRU
- Where
- 3 sites in Canada
- Volunteers
- 126 people
- Sponsor
- Baxalta now part of Shire
Atopic Dermatitis
Effectiveness of a Combined Dermatological Treatment in Children With Atopic Dermatitis
The purpose of this study is to determine the effectiveness of a combined dermatological treatment in children up to 5 years old with mild or moderated atopic dermatitis.
- Trial name
- KAOS (NCT02920411)
- Testing
- Pediatopic Treatment Cream, Pediatopic Body lotion
- Where
- 2 sites in Spain
- Volunteers
- 43 people
- Sponsor
- Laboratorios Ordesa
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks.
- Conditions
- To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disorders
- Trial name
- NCT04134299
- Testing
- AXA4010
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- Axcella Health, Inc
Non Small Cell Lung Cancer (NSCLC), Squamous Cell Carcinoma of the Head and Neck (SCCHN)
Study of PX-866 and Docetaxel in Solid Tumors
Phase 1: To determine the maximally tolerated dose (MTD) or recommended dose (RD) and any potential efficacy of PX-866 in combination with docetaxel in patients with solid tumors.
- Trial name
- NCT01204099
- Testing
- Docetaxel, PX-866
- Where
- 33 sites in United States, Canada
- Volunteers
- 223 people
- Sponsor
- Cascadian Therapeutics Inc.
Thyroid Eye Disease (TED)
A Study of VV-14305 for the Treatment of Thyroid Eye Disease
The goal of this study is to evaluate the safety, tolerability, and efficacy of KRIYA-586 (VV-14305) in treating thyroid eye disease (TED).
- Trial name
- RECLAIM (NCT07404111)
- Testing
- VV-14305
- Where
- 5 sites in United States, New Zealand
- Volunteers
- 135 people
- Sponsor
- Kriya Therapeutics, Inc.
Atopic Dermatitis
A Phase II Study in Adult Patients With Moderate to Severe Atopic Dermatitis
This proposed study is a randomized, double-blind, placebo-controlled, 3-arm parallel, multicenter phase II study, designed to explore the efficacy and safety of SHR0302 treatment for patients with moderate to severe atopic…
- Trial name
- NCT04162899
- Testing
- Drug: SHR0302
- Where
- 23 sites in China
- Volunteers
- 105 people
- Sponsor
- Reistone Biopharma Company Limited
Prader-Willi Syndrome
A Phase 3 Extension Study of RAD011 (Cannabidiol Oral Solution) in Patients With Prader-Willi Syndrome
This is a Phase 3 open-label extension (OLE) study in patients diagnosed with Prader-Willi Syndrome (PWS) who completed the Maintenance Period of the randomized, placebo-controlled Phase 2/3 study SCOUT-015.
- Trial name
- NCT05387798
- Testing
- RAD011
- Sponsor
- Radius Pharmaceuticals, Inc.
Atopic Dermatitis
Reconstitution With Pimecrolimus Cream 1% of Steroid-damaged Skin in Adults With Atopic Dermatitis
Topical steroid use may lead to skin deterioration and spider veins. This study will examine long-term management of atopic dermatitis (AD) over 12 months with pimecrolimus cream 1% and its effect on skin reconstitution of…
- Trial name
- NCT00367393
- Testing
- Pimecrolimus cream 1%
- Where
- Germany
- Volunteers
- 41 people
- Sponsor
- Novartis
Gout, Hyperuricemia
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABP-671 in Healthy Subjects
The purpose of this study is to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending doses of ABP-671 administered orally in healthy volunteers.
- Trial name
- NCT03906006
- Testing
- ABP-671
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Atom Therapeutics Co., Ltd
Alpha 1-Antitrypsin Deficiency
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency
The objective of this study is to prospectively assess whether there is any interval between first symptom and initial diagnosis that is experienced by patients with newly diagnosed alpha-1 antitrypsin deficiency (AATD) and then…
- Trial name
- NCT03039335
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Biocerna LLC
Sickle Cell Disease
Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients
This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease…
- Trial name
- NCT05791591
- Testing
- NUV001 - IR, NUV001 - GR
- Where
- 9 sites in India
- Volunteers
- 168 people
- Sponsor
- LGD
Absence Epilepsy, Jeavons Syndrome
BMB-101 in Absence Epilepsy and DEE
The study is a pilot, open-label, study to test whether BMB-101 is safe and effective in reducing the frequency of seizures in subjects with Absence Epilepsy including Epilepsy with Eyelid Myoclonia (also called Jeavons Syndrome)…
- Conditions
- Absence Epilepsy, Jeavons Syndrome, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- NCT06401538
- Testing
- BMB-101
- Where
- 5 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Cystic Fibrosis
Ease of Use and Microbial Contamination of Tobramycin Inhalation Powder (TIP) Versus Nebulised Tobramycin Inhalation Solution (TIS) and Nebulised Colistimethate (COLI)
The purpose of this interventional Phase IV study was to explore the ease of use of TIP and prevalence of microbial contamination of the T-326 Inhaler compared with TIS and colistimethate administered via nebuliser for the…
- Trial name
- NCT01844778
- Testing
- Tobramycin Inhalation Powder, Tobramycin inhalation solution, Colistimethate
- Where
- 21 sites in Germany, Ireland and 3 more
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin.
- Trial name
- NCT01987908
- Testing
- Aes-103
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- Baxalta now part of Shire
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
Coronary Artery Disease, Chest Pain
Coronary Obstruction Detection by Molecular Personalized Gene Expression (Corus CAD or ASGES)
To validate the use of Corus CAD (Age/Sex/Gene Expression score - ASGES) blood assay in subjects who are referred for the work-up of coronary artery disease.
- Conditions
- Coronary Artery Disease, Chest Pain, Cardiovascular Diseases, Coronary Heart Disease
- Trial name
- COMPASS (NCT01117506)
- Testing
- Corus CAD (ASGES)
- Where
- 7 sites in United States
- Volunteers
- 581 people
- Sponsor
- CardioDx
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